Eteplirsen fights to get accelerated approval status from the FDA

The US Food and Drug Administration Advisory Committee has voted against accelerated approval for eteplirsen, a drug designed to combat Duchenne muscular dystrophy (DMD). The drug was put up for accelerated approval under the FDA Safety and Innovation Act (FDASIA), but the committee found insufficient grounds for approval, noted Dr Nicola Davies in her monthly exclusive FDA Blog for The Pharma Letter.

The FDA Safety and Innovation Act (FDASIA)

The FDASIA is a piece of legislature, introduced in 2012, that aims to improve the efficiency of existing FDA regulatory processes. A major component of the act is the ability of the FDA to collect user fees to fund reviews of new and innovative drugs that companies are attempting to bring to market. The collection of these fees allows the FDA to increase the efficiency of the approval process, and also to fund research into the regulations governing the process. The aim is to make it easier and safer for the public to gain access to drugs in development.